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Gene therapy may cure rare diseases. But drugmakers have few incentives, leaving families desperate
CBS19 News Charlottesville· 8 minutes agoDrugmaker GlaxoSmithKline transferred another therapy to Orchard, which announced in 2019 that it had secured a designation from the U.S. Food and Drug Administration meant to speed up development and review.
Duchenne approval exposes FDA rift over Sarepta gene therapy
BioPharma Dive via Yahoo Finance· 12 hours agoPeter Marks’ decision to override the objections of agency staff and broaden use of Elevidys could...
Social media is heating up over why Asians don’t have body odor
NBC News via Yahoo News· 33 minutes agoSocial media users are using the heat wave as a chance to point out many Asians simply don’t smell....
Top FDA official overrules staff to approve gene therapy that failed trial
Ars Technica· 2 hours agoThe Food and Drug Administration (FDA) on Thursday announced expanded approval for a gene therapy to...
Family's genes offer clue to delaying onset of Alzheimer's
Napa Valley Register· 12 hours agoScientists studying a family plagued by early-in-life Alzheimer’s found some carry a genetic oddity...
Deadspin | Gene Haas to operate one of SHR’s Cup charters with new team
Deadspin· 23 hours agoStewart-Haas Racing will shut down after the 2024 NASCAR season, but Gene Haas isn't getting out of...
Takeaways from AP's report on access to gene therapies for rare diseases
Associated Press via Yahoo News· 19 hours agoSuch treatments offer the possibility of one-time cures. Collectively, about 350 million people...
FDA expands approval of first gene therapy for rare form of muscular dystrophy
CNN.com· 7 hours agoThe US Food and Drug Administration has given the green light for the first gene therapy that treats...
Rare gene mutation helps people resist Alzheimer’s disease - The Boston Globe
The Boston Globe· 1 day agoScientists studied her DNA and scanned her remarkable brain, ultimately tracing her protection to a...
Sarepta Catapults 30% After Hitting A 'Grand Slam' Approval In Muscular Dystrophy
Investor's Business Daily· 3 hours agoSarepta stock rocketed Friday after hitting a "grand slam" approval that expands the market for its...